Clinical Trials in the USA / NCT06924970
Active, not recruiting
Phase 2
A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD)
NCT06924970 · tracked via the Priya Life Science USA tracker
Condition(s) studied
Sickle Cell Disease
Investigational drug(s) / intervention(s)
Tebapivat →Tebapivat Matched Placebo
Tebapivat: Oral tablets.
Tebapivat Matched Placebo: Oral tablets.
Study summary
The main purpose of this study is to compare the effect of tebapivat versus placebo on anemia and to detect a dose-response for hemoglobin (Hb) response in participants with SCD.
Eligibility
Key Inclusion Criteria:
* Documented diagnosis of SCD (HbSS, HbSC \[combined heterozygosity for hemoglobins S and C\], sickle hemoglobin \[HbS\]/β0-thalassemia, HbS/β+-thalassemia, or other sickle cell syndrome variants).
* Hemoglobin ≥5.5 and ≤10.5 grams per decilitre (g/dL). Hemoglobin concentration must be based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the screening period.
* If taking hydroxyurea, the hydroxyurea dose must be stable for at least 90 days before randomization. Discontinuation of hydroxyurea requires a 90-day washout before providing informed consent.
Key Exclusion Criteria:
* Receiving regularly scheduled red blood cell (RBC) transfusion therapy (also termed chronic, prophylactic, or preventative transfusion); episodic transfusion in response to worsened anemia or vaso-occlusive crisis (VOC) is permitted. Additionally, a participant who requires episodic transfusion(s) may not have received a transfusion(s) within 60 days before providing informed consent or during the screening period.
* \>10 sickle cell pain crisis (SCPCs) in the 12 months before providing informed consent.
* Receiving anabolic steroids that have not been stopped for at least 4 weeks before randomization. Testosterone replacement therapy to treat hypogonadism is allowed; the testosterone dose and preparation must be stable for ≥10 weeks before randomization.
* Hospitalized for an SCPC and/or other vaso-occlusive event within 14 days before providing informed consent or within 14 days before randomization. If an SCPC occurs during the screening period, the screening period may be extended with Medical Monitor approval.
* Receiving treatment with voxelotor, crizanlizumab, or L-glutamine within 90 days before randomization.
* Platelet count \<lower limit of normal (LLN) for the local laboratory or \<150×109/liter (L) (whichever is lower) during screening. Platelet transfusions received within 28 days before consent or during screening.
* Receiving treatment with hematopoietic stimulating agents within 90 days before randomization.
* Prior exposure to gene therapy or prior bone marrow or stem cell transplantation, including any conditioning regimen.
Primary outcome measure(s)
- Percentage of Participants With Hb Response — Baseline, Week 10 through Week 12
Trial sites (20)
| Facility | City | Region | Status |
|---|---|---|---|
| UConn Health | Farmington | Connecticut | |
| MedStar Washington Hospital Center | Washington D.C. | District of Columbia | |
| Emory-Children's Center/ Children's Healthcare of Atlanta: Arthur M. Blank Hospital | Atlanta | Georgia | |
| Henry Ford Health System | Detroit | Michigan | |
| Children's Hospital of Michigan | Detroit | Michigan | |
| Icahn School of Medicine at Mt. Sinai | New York | New York | |
| University of Pittsburgh Medical Center | Pittsburgh | Pennsylvania | |
| Prisma Health Cancer Institute - Farris Road | Greenville | South Carolina | |
| University of Texas Health Science Center of Houston | Houston | Texas | |
| Fred Hutchinson Cancer Center, University of Washington | Seattle | Washington | |
| CHR de la Citadelle | Liège | Wallonne | |
| CHU Montreal | Montreal | Quebec | |
| Hôpital Edouard Herriot, CHU de Lyon | Lyon | Auvergne-Rhône-Alpes | |
| Institut Universitaire du Cancer de Toulouse - Oncopole | Toulouse | Midi Pyrenees | |
| CHU Hôpital Henri Mondor | Créteil | Île-de-France Region | |
| St. James Hospital | Dublin | Leinster | |
| Amsterdam Universitair Medisch Centrum, Locatie AMC | Amsterdam | North Holland | |
| Erasmus MC | Rotterdam | South Holland | |
| Universitair Medisch Centrum Utrecht | Utrecht | Netherlands | |
| University College London | London | United Kingdom |
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Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06924970 on ClinicalTrials.gov ↗ ← All trials in the USA